Williams: A bitter pill to swallow - who owns you?

Darcy Olsen, president of the Arizona-based Goldwater Institute, and Richard Garr, president of Neuralstem, a biotech company, wrote “Right to Try experimental drugs” in USA Today on May 28. They pointed out that “this year, more than 5,000 Americans will lose their battle with ALS, commonly known as Lou Gehrig’s disease.”

Up until recently, there was no medicine on the market that significantly improved the lives of ALS patients. But now there is one in clinical trials that holds considerable promise, but it has not been granted Food and Drug Administration approval. The average amount of time it takes to get a drug through the FDA approval process is 10 years. That’s time that terminal patients don’t have.

Legislators in Colorado, Louisiana and Missouri recently approved “Right to Try” legislation, and Arizona voters will vote on the measure this November. “Right to Try” is an initiative designed by the Goldwater Institute. It would give terminal patients access to investigational drugs that have completed basic safety testing. Under a doctor’s supervision, people would be given the chance to try promising experimental drugs before they’re given final FDA approval.

There’s no denying that there’s risk in taking a drug or medical procedure that hasn’t completed clinical trials. The question is: Who has the right to decide how much risk a person will take — he or some faceless Washington bureaucrat? In my opinion, the answer depends upon the answer to the question: Who owns you? If one owns himself, then it is he who decides how much risk he takes. If government owns you, then you don’t have the right to unilaterally decide how much risk you’ll take.

The FDA’s mission is to ensure the safety and effectiveness of pharmaceuticals. In doing so, FDA officials can make two types of errors. They can approve a drug that has unanticipated dangerous side effects, or they can disapprove or delay a drug that is both safe and effective. FDA officials have unequal incentives to avoid these two types of errors. If the FDA official errs on the side of under-caution — approving a dangerous drug — the victims are visible, and he is held directly accountable. If he errs on the side of over-caution — holding up approval of a safe and effective drug — who’s to know? The cost and the victims are invisible. Politicians and bureaucrats prefer invisible victims.

Here are a couple of notable examples. Clozapine was approved and used in 1972 in Europe. Clozapine’s ability to treat schizophrenics who did not respond to other medicines became well-known by 1979. Yet the drug was not approved in the United States until 1989 because companies believed that the FDA would reject it on the grounds that 1 percent of patients who took the drug contracted a blood disease. As an article in The New England Journal of Medicine stated, “what is remarkable is that clozapine has a beneficial effect in a substantial proportion (30 to 50 percent) of patients who have an inadequate response to other ... drugs.” Nearly 250,000 people with schizophrenia suffered needlessly, when relief was at hand.

According to Robert M. Goldberg, writing for the journal Regulation, “Mevacor is a cholesterol-lowering drug that has been linked to reduction in death due to heart attacks. It was available in Europe in 1989 but did not become available in the United States until 1992. Studies confirm what doctors saw to be the case: taking the drug reduces death due to heart disease by about 55 percent. During that three-year period as many as a thousand people a year died from heart disease because of the FDA delay.”

There is self-correction when a drug that has unanticipated dangerous side effects has been marketed. The drug is removed. But there’s no self-correction when a safe, effective lifesaving drug is not approved or is delayed. Those 5,000 ALS patients who will die of their disease this year are invisible, and FDA officials are unaccountable. “Right to Try” legislation is a step in the right direction to remedy that.

Walter E. Williams is a professor of economics at George Mason University.

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Of course, people should be allowed to take any medication they want for any reason they want, without the government interfering to insist on demonstrated safety and efficacy. There's no need for physicians to prescribe medicine, people can figure out for themselves what is in their best interest- after all, if they're wrong, it's they who will die. They costs of developing medicines will drop dramatically when manufacturers don't have to waste all that money on studies to establish dosing, safety and efficacy, and everything will be just perfect, like it was before the Food, Drug, and Cosmetic Act of 1938. ARE YOU CRAZY?

The example of Clozapine is misrepresented. The drug was introduced in Europe in 1972 and withdrawn from the market by the manufacturer in 1975 because of its severe (and sometimes fatal) side-effects. It was remarketed after governmental approvals in Europe and the U.S. in 1989 because new and better data on safety and efficacy became available.

A better example would have been thalidomide, marketed in Europe from 1957 to 1961 as a treatment for insomnia ands morning sickness. By the time it was withdrawn in 1961, there had been 10,000 to 20,000 babies born with birth defects related to the drug, and half of them died. The drug had never been tested in pregnancy, either in animals or humans, prior to marketing. The manufacturer asked two American companies to market the medication, and one agreed but was refused six times by the FDA because there was no data on safety in pregnancy. Clinical trials were begun but went on only as few months before being stopped because of the recognized risks occurring elsewhere in the world. Only 17 malformed babies were born to trial participants. That's exactly how the FDA is supposed to work.

It is absolutely true that development, testing and approval of a new drug is a very long and costly process, and some drugs are not developed because not enough revenue could be recouped. Most are "me too" drugs closely related to drugs already available, but some are novel treatments for diseases which affect relatively small numbers of patients. There are federal funds to support such drugs, called "orphan drugs". Every drug under development also has clinical trials available for people who fit specific parameters of a test group, and all have protocols for "compassionate use," to make it available to those who don't quite fit an open trial or in the gap between completing trials and obtaining approval.

There has been a lot of junk rejected by the FDA (like laetrile, the Mexican apricot pit extract for cancer) and plenty of good drugs sent back for more safety or efficacy data after which they were approved. I don't know any "good" drug rejected that never came back and got approval. Do we have a pharmacist among our readers?

With these "Right to Try" initiatives passed in CO, LA, & MO, hopefully there's language in the law that severely limits or doesn't allow lawsuits to be filed against the pharmaceutical companies. I think Bubba makes some good points but if someone is willing to try an experimental drug and has been given the most current information available and is under the care of a physician, then I think they should be able to try it. Just know that with a risky proposition comes risky consequences (good or bad) and in the end - the buck stops with the consumer (in this case of trying experimental drugs).

Let me get this right, people are allowed or soon will be allowed to use medical marijuana without FDA approval? People are moving, complaining and demanding medical marijuana with no real proof of the safety in children? Next, as prescriptions are passed out like candy and more elderly people become drug addicts by prescription, we will not let a willing adult patient try a unapproved drug? What is wrong with this picture? No one, absolutely no one is suggesting that policy and requirements be shortened as implied. We let a grown man who can afford it drive a Corvette or Porsche but we will not let one try an untested drug? We will let a grown man choose to wear or not wear a motorcycle helmet but he cannot try a new drug? While at the same time commercials run for Testosterone therapy and the the law suits against it ? Now that is hypocrisy !

Note: I would add ADD/ADHD drugs to this note but each time I do, someone gets offended!

I am not ! Two semesters ago, I took Psychopharmacology at A/C under Dr Banks. That along with several other Pharmacology classes in years past, I have a pretty good ideal about the content and structure of some drugs both licit and illicit. Over the counter drugs, cold meds and pseudoephedrine are destroying more lives, more livers and more kidneys than any unapproved drugs or illegal drugs. The " stuff " that any kid, any adult can walk into CVS or Target and buy right off the shelf is destroying the human body & possibly causing the allergies and illness. Next, super strength antibiotics, Z Packs are destroying the immune system and no one is complaining about the health and well being of young patients. Last, is .... you know what, never mind. Your civic leaders, college professors and elected officials are telling you all " it's all ok "

When I asked if we have a pharmacist among our readers, I meant an actual pharmacy school graduate, licensed by the state, not a former drug abuser who has taken a couple of courses at the community college to obtain some sort of counseling or rehabilitation credential but couldn't find his aspirin with both hands and a flashlight (if you don't get the joke, I'll be happy to explain it). Charles, is there anything about which you don't know more than anyone else?